- Introducing first-in-human therapies safely in small patient populations
- Balancing safety, efficacy and speed throughout development
- Using adaptive clinical trial designs where appropriate to accelerate development without compromising quality
- Working effectively with orphan drug pathways and regulators
- Practical lessons from rare disease clinical development programmes that delegates can apply within their own organisations
- Identifying high-risk protocol elements before finalisation
- Using site and investigator feedback before study start-up
- Balancing scientific ambition with operational feasibility
- Reducing rework through better cross-functional review
- Identifying where AI is already improving diagnosis
- Integrating AI into existing clinical workflows
- Overcoming implementation and governance challenges
- Measuring impact on referral pathways and patient outcomes
- Understanding why patients discontinue treatment or follow-up
- Educating patients on the value of remaining in clinical trials
- Balancing long-term study requirements with patients’ treatment opportunities
- Designing studies that support both retention and patient choice
